STAT+: FDA approves first treatment for skin condition that causes persistent wounds, a redosable gene therapy
The FDA approved the first treatment for dystrophic epidermolysis bullosa, a devastating condition that causes the skin to be extraordinarily fragile.
The Food and Drug Administration on Friday approved the first treatment for a devastating condition that causes the skin to be so fragile that even a touch can cause it to splinter, bringing another gene therapy onto the market.
The therapy, known as Vyjuvek and made by Krystal Biotech, will soon be available for patients with dystrophic epidermolysis bullosa, a genetic disease that causes painful blisters and persistent wounds. Patients with the condition live covered in bandages to try to stop new blisters from forming and to dress their wounds. Scarring can cause fingers and toes to fuse together, and patients face high rates of skin cancer.
Vyjuvek, a topical treatment, works by delivering a healthy copy of the gene that encodes the protein type VII collagen to the targeted skin cells. The protein anchors the layers of skin together and enables healing to occur. With the mutated form of the protein that patients make, the slightest friction can cause a blister to form, and the skin has trouble closing the resulting wounds.
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